Evaluating XH-S003 Capsules for Patients with Paroxysmal Nocturnal Hemoglobinuria

A Multicenter, Randomized, Single-blind Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

This study is testing if XH-S003 capsules can help people with Paroxysmal Nocturnal Hemoglobinuria feel better and if they have any side effects.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: S-INFINITY Pharmaceuticals Co., Ltd (Industry-sponsored)
• Locations: 1 site (Tianjin)
• Trial ID: NCT06978699 on ClinicalTrials.gov

What this trial studies

This Phase II trial is a multicenter, randomized, single-blind study aimed at assessing the efficacy and safety of XH-S003 capsules in patients diagnosed with Paroxysmal Nocturnal Hemoglobinuria (PNH). Approximately 24 participants will be enrolled and assigned to one of three different dose levels of the oral medication. The study will monitor the patients for improvements in their condition and any potential side effects associated with the treatment.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older who have been diagnosed with PNH and meet specific clinical criteria.

Not a fit: Patients who have previously received complement inhibitor therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients suffering from PNH, potentially improving their quality of life.

How similar studies have performed: While this approach is being evaluated in this trial, similar studies have shown promise in treating PNH with other therapies, indicating potential for success.

Eligibility criteria

Inclusion Criteria:

* Male or female with aged ≥18 years old;
* Weight ≥40 kg and BMI≥18 kg/m2 ;
* Diagnosed with PNH: with red blood cell or granulocyte clone levels \>10% detected by flow cytopy within 6 months prior to screening or during screening;
* Patients who have not previously received any complement inhibitor therayp;
* LDH \> 1.5×ULN detected two times during the screening period (interval of 2 to 8 weeks);
* Hb meets one of the following conditions: (1) Hb \<100 g/L at the first screening visit, and subjects receive RBC transfusion because of PNH-related anemia during the screening period; (2) The average Hb of two tests during the screening period \<100 g/L (interval of 2\~8 weeks);
* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae before the first administration. If the subject has not been vaccinated previously or requires booster vaccination (according to local vaccination policies), vaccination must be administered at least 2 weeks before the first administration. If the first administration must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination;

Exclusion Criteria:

* Subjects with laboratory evidence of bone marrow failure during the screening period (reticulocyte count \<100×109/L, platelet count \<30×109/L, or neutrophil count \<0.5×109/L);
* Subjects receiving other therapies prior to screening who have not achieved the following treatment durations:

  • Erythropoietin or immunosuppressants for at least 8 weeks; • Systemic corticosteroids for at least 4 weeks; • Iron supplements, vitamin B12, or folic acid for at least 4 weeks; • Anticoagulants: Vitamin K antagonists for at least 4 weeks with stable international normalized ratio (INR) (as determined by the investigator), low molecular weight heparin for at least 4 weeks; • Hypoxic-inducing factor prolyl hydroxylase inhibitors (HIF-PHI) for at least 8 weeks; • Androgens for at least 4 weeks;
* A history of bone marrow/hematopoietic stem cell or solid organ transplantation;
* Alanine aminotransferase (ALT), γ-glutamyl transpeptidase (GGT), or alkaline phosphatase (ALP) \>3×ULN at screening; - Positive HIV antibody, active syphilis infection, positive HBsAg, active HCV infection, or active tuberculosis infection at screening;
* Known or suspected immunodeficiency diseases or hereditary complement deficiency at screening;
* A history of Neisseria meningitidis infection;
* Subjects with chronic active or recurrent infections within 1 year prior to screening;
* Subjects with systemic active bacterial, viral (including COVID-19), or fungal infections within 2 weeks prior to the first administration; subjects with body temperature \>38°C within 7 days prior to the first administration;

Where this trial is running

Tianjin

• TianJin Medical University General Hospital — Tianjin, China (Recruiting)

Study contacts

• Study coordinator: Rong Fu

Conditions PNH - Paroxysmal Nocturnal Hemoglobinuria; Paroxysmal Nocturnal Hemoglobinuria